Recent Updates
Recently added Catalysts

REGN7999

Phase 2

Non-Transfusion Dependent Beta-Thalassemia (NTDT) | Small molecule | Hematology |Regeneron Pharmaceuticals, Inc.|Last Updated: Jul 1, 2026

Success Probability
Subscribe to view
Market & Valuation
Subscribe to view
Trial Design
RandomizedDouble-BlindPLACEBO_CONTROLLED
Total Trials1
Total Enrollment19
FDA Designations
No designations recorded
Clinical trial landscape

REGN7999 · 2 trials · 2 indications

Phase 2 1Phase 1 1
NCT06421636A Study to Test the Safety, Tolerability, and Efficacy of an Antibody, REGN7999, Injected Under the Skin for the Treatment of Iron Overload in Adult Participants With Non-Transfusion Dependent β-thalassemia, Using MRI Scans to Measure Iron Levels in the BodyNon-Transfusion Dependent Beta-Thalassemia (NTDT)
ACTIVE NOT_RECRUITING19 Analytics
PHASE2ACTIVE NOT_RECRUITING
A Study to Test the Safety, Tolerability, and Efficacy of an Antibody, REGN7999, Injected Under the Skin for the Treatment of Iron Overload in Adult Participants With Non-Transfusion Dependent β-thalassemia, Using MRI Scans to Measure Iron Levels in the Body
Non-Transfusion Dependent Beta-Thalassemia (NTDT)
NCT0642163619
Unlock trial analytics
Study Endpoints
Primary Endpoints
Change from baseline in Liver Iron Concentration (LIC) by R2* Magnetic Resonance Imaging (MRI)
At week 24
Incidence of Treatment-Emergent Adverse Events (TEAEs)
Up to week 72
Severity of TEAEs
Up to week 72
Incidence and severity of treatment emergent adverse events (TEAEs) in participants treated with REGN7999 or placebo
Through the end of study visit, week 20

IV cohorts 1 to 4 SC cohorts 1 and 2

Incidence and severity of TEAEs in participants treated with REGN7999 or placebo
Through the end of study visit, week 26

IV cohort 5 and SC cohort 3

Secondary Endpoints
Achievement of ≥20% reduction from baseline in LIC by R2* MRI
Through week 52
Change from baseline in hemoglobin
To week 24
Change from baseline in LIC by R2* MRI
At week 52
Unlock Study Endpoints
Study Design & Arms
AllocationRANDOMIZED
MaskingQUADRUPLE
ModelPARALLEL
PurposeTREATMENT
Treatment Arms
ArmTypeDescription
Part A High DoseEXPERIMENTALRandomized 2:2:1
Part A Low DoseEXPERIMENTALRandomized 2:2:1
Part A PlaceboPLACEBO_COMPARATORRandomized 2:2:1
Part B High DoseEXPERIMENTALRandomized 2:2:1
Part B Low DoseEXPERIMENTALRandomized 2:2:1
Part B PlaceboPLACEBO_COMPARATORRandomized 2:2:1
IV Cohort 1EXPERIMENTALSingle dose REGN7999 or Placebo; randomized 3:1
IV Cohort 2EXPERIMENTALSingle dose REGN7999 or Placebo; randomized 3:1
IV Cohort 3EXPERIMENTALSingle dose REGN7999 or Placebo; randomized 3:1
IV Cohort 4EXPERIMENTALSingle dose REGN7999 or Placebo; randomized 3:1
IV Cohort 5EXPERIMENTALSingle dose REGN7999 or Placebo; randomized 3:1
SC Cohort 1EXPERIMENTALSingle dose REGN7999 or Placebo; randomized 3:1
SC Cohort 2EXPERIMENTALSingle dose REGN7999 or Placebo; randomized 3:1
SC Cohort 3EXPERIMENTALSingle dose REGN7999 or Placebo; randomized 3:1
Interventions
NameTypeDescription
REGN7999DRUGAdministered subcutaneous (SC)
PlaceboDRUGAdministered SC
Unlock Study Design Details
Eligibility Criteria
Age Range18 Years to 65 Years
SexALL
Healthy VolunteersNo
Study Sites26

Key Inclusion Criteria: 1. Clinical diagnosis of NTDT as described in the protocol 2. IOL, defined as LIC ≥ 5 mg Fe/g DW as measured by R2\* MRI at screening 3. Serum ferritin ≥ 300 ng/mL as described in the protocol Key Exclusion Criteria: 1. Hemoglobin ≤ 8 g/dL at screening 2. Any RBC transfusi...

Countries:United StatesGeorgiaGreeceIndiaItalyMalaysiaThailandTurkey (Türkiye)United KingdomBelgium
Unlock Eligibility Criteria
Recent Changes (Last 90 Days)
HIGHJul 2, 2026NCT06421636Status: RECRUITING → ACTIVE_NOT_RECRUITING
HIGHJul 2, 2026NCT06421636Status: RECRUITING → ACTIVE_NOT_RECRUITING
HIGHJul 2, 2026NCT06421636Status: RECRUITING → ACTIVE_NOT_RECRUITING
HIGHJul 2, 2026NCT06421636Status: RECRUITING → ACTIVE_NOT_RECRUITING
LOWMay 26, 2026NCT06421636primaryCompletionDate: changed
LOWMay 24, 2026NCT06421636studyFirstPostDate: changed